The main purpose of this study is to determine the safety and tolerability of three different doses of AT2220 in people affected by Pompe disease. The study will also evaluate the effects of AT2220 on functional parameters in Pompe disease.

Subjects of reproductive potential agree to use reliable methods of contraception during the study

Subject or legal representative is willing and able to provide written informed consent

Exclusion Criteria:

Any intercurrent condition that may preclude accurate interpretation of study data

Obstructive pulmonary disease

Invasive ventilatory support

Use of noninvasive ventilatory support >8 hours/day while awake

History of QTc prolongation >450 msec for males and >470 msec for females

History of allergy or sensitivity to the study drug, including any prior serious adverse reaction to iminosugars (e.g., miglustat, miglitol)

Pregnancy or breast-feeding

Current or recent drug or alcohol abuse

Treatment with another investigational drug within 30 days of study start

Use of prohibited medications ≤3 months prior to screening

Otherwise unsuitable for the study in the opinion of investigator

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00688597